Low-cost method may quickly spot carriers of SMA-causing mutations
Researchers in China report having developed a way to quickly and more easily screen for people who carry SMN1 gene…
Articles by Andrea Lobo
1 in 59 women in region of China found to be SMA carrier: Study
Among women of reproductive age in a southern region of China, one in every 59 tested in a screening program…
Report highlights poor bone health, leg fracture risk in child with SMA
The case of a 4.5-year-old girl with spinal muscular atrophy (SMA) type 2 who had acute pain…
Voyager, Novartis join to advance next-generation SMA gene therapy
Voyager Therapeutics is collaborating with Novartis to discover and develop next-generation gene therapies for conditions like…
Gene editing of SMN2 leads to normal SMN protein levels: Study
Researchers in the U.S. report using a gene-editing method to alter the SMN2 gene sequence, leading to normal levels of…
Revvity launches platform for streamlined newborn screening
Revvity has launched its EONIS Q system for faster and simplified newborn molecular screening of spinal muscular…
Oral SBMA therapy shows safety and tolerability in Phase 1 trial
NIDO-361, Nido Biosciences’ experimental oral treatment for spinal and bulbar muscular atrophy (SBMA), was seen to be…
Bone health is impaired in SMA children, but treatment may help
Children with spinal muscular atrophy (SMA) generally have poor bone health, including low bone mineral density and reduced bone…
Spinraza treatment responses may be predicted by certain RNAs: Study
Certain RNA molecules may be used as biomarkers to assess response to treatment with Spinraza (nusinersen) in people with…
SMA may affect some cognitive abilities in type 2 and 3 children
Intellectual disability is not common among children with type 2 and 3 spinal muscular atrophy (SMA), but some aspects…